Nature of Business

12 Months Ended

Dec. 31, 2020

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Nature of Business

Ocugen, Inc., together with its wholly owned subsidiaries (“Ocugen” or the “Company”), is a biopharmaceutical company focused on developing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. The Company is located in Malvern, Pennsylvania.

Ocugen is co-developing COVAXIN, a whole-virion inactivated COVID-19 vaccine candidate, with Bharat Biotech International Limited ("Bharat Biotech") for the U.S. market. COVAXIN is being developed to prevent COVID-19 infection in humans and is formulated with the inactivated SARS-CoV-2 virus, an antigen, and an adjuvant. In February 2021, the Company entered into a Co-Development, Supply and Commercialization Agreement (the "Covaxin Agreement") with Bharat Biotech, pursuant to which the Company obtained an exclusive right and license under certain of Bharat Biotech's intellectual property rights, with the right to grant sublicenses to develop, manufacture, and commercialize COVAXIN for the prevention of COVID-19 in humans in the United States, its territories and possessions (the “Ocugen Covaxin Territory”). COVAXIN has been granted approval for emergency use in India. A Phase 3 clinical trial is ongoing in India. The Company is currently evaluating the clinical and regulatory path for COVAXIN in the United States including obtaining Emergency Use Authorization ("EUA") from the U.S. Food and Drug Administration (the "FDA") and, eventually, biologic license application (“BLA”) approval in the U.S. market, as well as the Company's commercialization strategy, if authorized or approved. See Note 16 for additional information about the terms, rights, and obligations under the Covaxin Agreement.

Ocugen is developing a breakthrough modifier gene therapy platform to generate therapies designed to fulfill unmet medical needs in the area of retinal diseases, including inherited retinal diseases ("IRDs") and dry age-related macular degeneration ("AMD"). Ocugen's modifier gene therapy platform is based on nuclear hormone receptors (“NHRs”), which have the potential to restore homeostasis, the basic biological processes in the retina. Unlike single-gene replacement therapies, which only target one genetic mutation, Ocugen believes that its gene therapy platform, through its use of NHRs, represents a novel approach in that it may address multiple retinal diseases with one product.

OCU400 is the Company's first product candidate being developed with the Company's modifier gene therapy platform. OCU400 is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse IRDs, including retinitis pigmentosa ("RP") and leber congenital amaurosis ("LCA"). OCU400 has received four Orphan Drug Designations from the FDA for the treatment of certain disease genotypes: nuclear receptor subfamily 2 group E member 3 ("NR2E3"), centrosomal protein 290 ("CEP290"), rhodopsin ("RHO"), and phosphodiesterase 6B ("PDE6ß") mutation-associated inherited retinal degenerations. Ocugen is planning to initiate two Phase 1/2a clinical trials for OCU400 in the United States in the second half of 2021. OCU400 additionally received Orphan Medicinal Product Designation from the European Commission, based on the recommendation of the European Medicines Agency, for RP and LCA in February 2021, which Ocugen believes further supports the broad spectrum application of OCU400 to treat many IRDs. Ocugen is currently evaluating options to commence OCU400 clinical trials in Europe in 2022. Ocugen's second gene therapy candidate, OCU410, is being developed to utilize the nuclear receptor genes RAR-related orphan receptor A ("RORA") for the treatment of dry AMD. This candidate is currently in preclinical development. Ocugen is planning to initiate a Phase 1/2a clinical trial for OCU410 in 2022.

Ocugen is also conducting preclinical development for its biologic product candidate, OCU200. OCU200 is a novel fusion protein designed to treat diabetic macular edema, diabetic retinopathy, and wet AMD. Ocugen had a pre-Investigational New Drug ("IND") meeting with the FDA in November 2020 and received guidance on IND-enabling preclinical studies to support the Phase 1/2a study. Ocugen expects to initiate IND-enabling preclinical studies for OCU200 in 2021. Ocugen plans to initiate a Phase 1/2a clinical trial for OCU200 in 2022.

Ocugen was developing OCU300, a small molecule therapeutic for the treatment of symptoms associated with ocular graft-versus-host disease. The Phase 3 clinical trial for OCU300 was discontinued in 2020 based on results of a pre-planned interim sample size analysis conducted by an independent Data Monitoring Committee, which indicated the trial was unlikely to meet its co-primary endpoints upon completion. Ocugen is no longer pursuing the development of this product candidate.

Merger with Histogenics

On September 27, 2019, the Company, which was formerly known as Histogenics Corporation ("Histogenics"), completed a reverse merger (the "Merger") with Ocugen OpCo, Inc. ("OpCo") in accordance with the terms of the Agreement and Plan of

Merger and Reorganization, dated as of April 5, 2019, by and among OpCo, Restore Merger Sub, Inc., the Company's wholly owned subsidiary ("Merger Sub"), and the Company, as amended (the "Merger Agreement") pursuant to which Merger Sub merged with and into OpCo, with OpCo surviving as the Company's wholly owned subsidiary. Immediately after completion of the Merger, the Company changed its name to Ocugen, Inc. and the business previously conducted by OpCo became the business conducted by the Company. OpCo is deemed to be the accounting acquirer. Accordingly, the historical financial statements of OpCo became the Company’s historical financial statements, including the comparative prior periods. See Note 3 for additional information.

Going Concern Consideration

The audited consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) assuming the Company will continue as a going concern. As of December 31, 2020, the Company had cash, cash equivalents, and restricted cash of approximately $24.2 million, and since that date the Company has received net proceeds of $4.8 million from the sale of the Company's common stock in an at-the-market offering (“ATM") commenced in August 2020 and net proceeds of $21.2 million from the sale of the Company's common stock in a registered direct offering (the "Registered Direct Offering"). See Note 9 for additional information about the August 2020 ATM. See Note 16 for additional information about the Registered Direct Offering. As a result of the Company's cash, cash equivalents, and restricted cash balance as of December 31, 2020 and the net proceeds received subsequent to December 31, 2020 from the August 2020 ATM and the Registered Direct Offering, the Company believes that its cash, cash equivalents, and restricted cash will enable the Company to fund its operating expenses and capital expenditure requirements through at least one year from the date the audited consolidated financial statements are issued.